Genetic Determinants of Amyotrophic Lateral Sclerosis as Therapeutic Targets

Author(s): Daryl A. Bosco, John E. Landers.

Journal Name:CNS & Neurological Disorders - Drug Targets

Volume 9 , Issue 6 , 2010

Abstract:

Amyotrophic lateral sclerosis (ALS) is an incurable disease resulting from the deterioration of motor neurons. The onset of disease typically occurs in the fifth decade of life and progresses rapidly; death occurs for 75% of patients within 5 years. The only drug that is available to treat ALS is riluzole, which extends survival by just 2-3 months. Thus, new therapeutic directions are being sought to prolong the lifespan of ALS patients. Since the discovery of SOD1 as a genetic determinant of ALS in 1993, SOD1-models of ALS have been extensively employed for the development of ALS therapeutics. Novel genetic targets are now under investigation following the recent discoveries linking TDP-43, FUS/TLS, angiogenin, KIFAP3 and UNC13A to ALS. In this review, we present several of the genetic contributors to both sporadic and familial forms of ALS and discuss their potential as therapeutic targets for this devastating disease.

Keywords: Amyotrophic lateral sclerosis, neurodegeneration, therapy, genome-wide association, SOD1, TDP-43, FUS/TLS, angiogenin, KIFAP3, UNC13A, familial ALS, Alzheimer's disease, Parkinson's disease, Huntington's disease, immunotherapy, FALS, chaperones, arimoclomol, TAR, SALS, frontotemporal lobar degeneration, (GWAS), DPP6

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Article Details

VOLUME: 9
ISSUE: 6
Year: 2010
Page: [779 - 790]
Pages: 12
DOI: 10.2174/187152710793237494
Price: $58

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